A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis

This study is looking for adults who are 18 years old or older. There is no upper age limit, so people of all adult ages are welcome to consider taking part.

Participants must have a confirmed diagnosis of either Alport Syndrome or a specific type of kidney scarring called Primary Steroid-Resistant Focal Segmental Glomerulosclerosis (FSGS).

If you have Alport Syndrome, you must also be currently taking standard medications (like ACE inhibitors or ARBs) but still have too much protein in your urine (this is called 'uncontrolled proteinuria'). This shows that your current treatment isn't fully managing the protein leak, and you might benefit from additional help.

If you join this study, you will be given the study medicine, R3R01, as part of the research. You will have regular visits to the clinic where the study team will carry out various checks. These will include physical examinations, measuring your vital signs (like blood pressure and heart rate), taking ECGs (heart tracings), and doing blood and urine tests. These tests help the researchers understand how your body is reacting to the medicine, if it's helping, and if there are any side effects. The study will measure the amount of protein in your urine after 12 weeks to see if the medicine is making a difference. There will also be follow-up appointments after you finish taking the medicine to ensure your health is stable. We do not have the exact number of visits or the total duration, but research studies typically involve multiple visits over several months.